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Molecular Therapies for Inherited Retinal Diseases—Current Standing, Opportunities and Challenges

Inherited retinal diseases (IRDs) are both genetically and clinically highly heterogeneous and have long been considered incurable. Following the successful development of a gene augmentation therapy for biallelic RPE65-associated IRD, this view has changed. As a result, many different therapeutic a...

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Bibliografski detalji
Izdano u:Genes (Basel)
Glavni autori: Vázquez-Domínguez, Irene, Garanto, Alejandro, Collin, Rob W. J.
Format: Artigo
Jezik:Inglês
Izdano: MDPI 2019
Teme:
Online pristup:https://ncbi.nlm.nih.gov/pmc/articles/PMC6770110/
https://ncbi.nlm.nih.gov/pubmed/31466352
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/genes10090654
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